The first 5years of clinical experience with laronidase enzyme replacement therapy for mucopolysaccharidosis I
Author:
Publisher
Informa Healthcare
Subject
Pharmacology (medical),Pharmacology,General Medicine
Link
http://www.tandfonline.com/doi/pdf/10.1517/14656566.6.3.489
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1. Advances in the treatment of lysosomal storage disease
2. α-L-Iduronidase and enzyme replacement therapy for mucopolysaccharidosis I
3. Enzyme replacement therapy for the mucopolysaccharide storage disorders
4. Mutational analysis of 85 mucopolysaccharidosis type I families: frequency of known mutations, identification of 17 novel mutations and in vitro expression of missense mutations
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